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2018年欧洲基因与细胞疗法协会（ESGCT）：RP-L201及RP-L401基因疗法的临床前数据

2018-10-21 MedSci MedSci原创

RP-L201RP-L401基因疗法
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RP-L201是一种基于LVV的LAD-I基因疗法，其携带编码Beta-2整合素组分CD18的ITGB2基因。CD18是一种关键蛋白，可促进白细胞粘附，并使其从血管中渗出以抵抗感染。RP-L201的全面安全性和有效性临床前研究显示小鼠模型中的稳定植入和表型校正，RP-L201具有恢复的中性粒细胞迁移的能力。

RP-L201是一种基于LVV的LAD-I基因疗法，其携带编码Beta-2整合素组分CD18的ITGB2基因。CD18是一种关键蛋白，可促进白细胞粘附，并使其从血管中渗出以抵抗感染。RP-L201的全面安全性和有效性临床前研究显示小鼠模型中的稳定植入和表型校正，RP-L201具有恢复的中性粒细胞迁移的能力。

RP-L401是一种基于LVV的IMO基因治疗，携带TCIRG1基因。TCIRG1基因的突变导致破骨细胞功能无效和骨吸收受损，导致IMO患者骨骼的异常生长，使神经系统异常畸形和骨髓衰竭。这种疾病在早期通常是致命的。RP-L401的临床前研究在TCIRG1突变的oc / oc小鼠模型中进行，在小鼠出生后不久给予的RP-L401，能够使大多数小鼠长期存活，使骨吸收的恢复和骨硬化表型的逆转成为可能。LAD-I和IMO基因疗法的积极临床前结果显示基因疗法治疗罕见病的潜力。


原始出处：

http://www.firstwordpharma.com/node/1598682#axzz5UXJIsme7

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#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1797293, encodeId=d3121e97293eb, content=<a href='/topic/show?id=a51422e0639' target=_blank style='color:#2F92EE;'>#临床前数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22706, encryptionId=a51422e0639, topicName=临床前数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=845e216, createdName=cqlidoudou, createdTime=Fri Jun 07 16:47:00 CST 2019, time=2019-06-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993717, encodeId=e4241993e1780, content=<a href='/topic/show?id=e962e888c0' target=_blank style='color:#2F92EE;'>#GCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7888, encryptionId=e962e888c0, topicName=GCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d7532500500, createdName=12499e01m47暂无昵称, createdTime=Mon Feb 18 14:47:00 CST 2019, time=2019-02-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1793760, encodeId=5c321e937608d, content=<a href='/topic/show?id=d86d1621546' target=_blank style='color:#2F92EE;'>#SGCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=40, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16215, encryptionId=d86d1621546, topicName=SGCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=9b30126, createdName=skhzy, createdTime=Mon Dec 31 09:47:00 CST 2018, time=2018-12-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339133, encodeId=5e6213391333b, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Tue Oct 23 08:47:00 CST 2018, time=2018-10-23, status=1, ipAttribution=)]
    2019-06-07 cqlidoudou

    #临床前数据#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1797293, encodeId=d3121e97293eb, content=<a href='/topic/show?id=a51422e0639' target=_blank style='color:#2F92EE;'>#临床前数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22706, encryptionId=a51422e0639, topicName=临床前数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=845e216, createdName=cqlidoudou, createdTime=Fri Jun 07 16:47:00 CST 2019, time=2019-06-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993717, encodeId=e4241993e1780, content=<a href='/topic/show?id=e962e888c0' target=_blank style='color:#2F92EE;'>#GCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7888, encryptionId=e962e888c0, topicName=GCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d7532500500, createdName=12499e01m47暂无昵称, createdTime=Mon Feb 18 14:47:00 CST 2019, time=2019-02-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1793760, encodeId=5c321e937608d, content=<a href='/topic/show?id=d86d1621546' target=_blank style='color:#2F92EE;'>#SGCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=40, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16215, encryptionId=d86d1621546, topicName=SGCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=9b30126, createdName=skhzy, createdTime=Mon Dec 31 09:47:00 CST 2018, time=2018-12-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339133, encodeId=5e6213391333b, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Tue Oct 23 08:47:00 CST 2018, time=2018-10-23, status=1, ipAttribution=)]
    2019-02-18 12499e01m47暂无昵称

    #GCT#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1797293, encodeId=d3121e97293eb, content=<a href='/topic/show?id=a51422e0639' target=_blank style='color:#2F92EE;'>#临床前数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22706, encryptionId=a51422e0639, topicName=临床前数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=845e216, createdName=cqlidoudou, createdTime=Fri Jun 07 16:47:00 CST 2019, time=2019-06-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993717, encodeId=e4241993e1780, content=<a href='/topic/show?id=e962e888c0' target=_blank style='color:#2F92EE;'>#GCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7888, encryptionId=e962e888c0, topicName=GCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d7532500500, createdName=12499e01m47暂无昵称, createdTime=Mon Feb 18 14:47:00 CST 2019, time=2019-02-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1793760, encodeId=5c321e937608d, content=<a href='/topic/show?id=d86d1621546' target=_blank style='color:#2F92EE;'>#SGCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=40, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16215, encryptionId=d86d1621546, topicName=SGCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=9b30126, createdName=skhzy, createdTime=Mon Dec 31 09:47:00 CST 2018, time=2018-12-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339133, encodeId=5e6213391333b, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Tue Oct 23 08:47:00 CST 2018, time=2018-10-23, status=1, ipAttribution=)]
    2018-12-31 skhzy

    #SGCT#

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1797293, encodeId=d3121e97293eb, content=<a href='/topic/show?id=a51422e0639' target=_blank style='color:#2F92EE;'>#临床前数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22706, encryptionId=a51422e0639, topicName=临床前数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=845e216, createdName=cqlidoudou, createdTime=Fri Jun 07 16:47:00 CST 2019, time=2019-06-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993717, encodeId=e4241993e1780, content=<a href='/topic/show?id=e962e888c0' target=_blank style='color:#2F92EE;'>#GCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7888, encryptionId=e962e888c0, topicName=GCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d7532500500, createdName=12499e01m47暂无昵称, createdTime=Mon Feb 18 14:47:00 CST 2019, time=2019-02-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1793760, encodeId=5c321e937608d, content=<a href='/topic/show?id=d86d1621546' target=_blank style='color:#2F92EE;'>#SGCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=40, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16215, encryptionId=d86d1621546, topicName=SGCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=9b30126, createdName=skhzy, createdTime=Mon Dec 31 09:47:00 CST 2018, time=2018-12-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1339133, encodeId=5e6213391333b, content=<a href='/topic/show?id=447663054f5' target=_blank style='color:#2F92EE;'>#欧洲#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63054, encryptionId=447663054f5, topicName=欧洲)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKxsKDoOOFxGpMibw6lsQFUIicWt7kWGevkdgFhjiban1icD2JdIBmjVo7qX7aSlCSpiaia382nuLKiaI3Cg/132, createdBy=5c442500150, createdName=ms9474365431325850, createdTime=Tue Oct 23 08:47:00 CST 2018, time=2018-10-23, status=1, ipAttribution=)]
    2018-10-23 ms9474365431325850

    #欧洲#

    0